Time has run short for taking a comprehensive look at one of the most difficult topics in the industry—how best to regulate compassion.

The regulatory approval of the first drug for Duchenne muscular dystrophy is shedding new light on existing controversies in orphan drug development. As we sift through ongoing media coverage, we wanted to highlight two key stories and their implications:

The Sarepta Dilemma: Bioethics Expert Arthur Caplan Says It’s Time to Rethink How to Regulate Compassion
EndPoints News
Oct. 10, 2016

Prominent bioethicist Arthur Caplan is calling for an overhaul of expanded access regulations and policies. In an interview with Endpoints, an online news service launched by the former editor of Fierce Biotech, Caplan argues: “We’re trying to balance the challenge of compassionate use against approval and I think we have to revisit the whole subject.


  • It’s important for companies to define and share compassionate use and expanded access policies early, before they’re needed.
  • Educating patient organizations and communities on these policies can help to manage expectations.
  • Involving credible third-party experts and organizations can provide additional context, resources, and constructive opportunities for stakeholders to get involved.

Wrong Again: Why Sarepta’s $300K Price for DMD Drug Invalidates Reasons for Accelerated Approval
Sept. 26, 2016

This lengthy editorial chastises Sarepta for its pricing of eteplirsen, saying “it is simply unconscionable to demand that public and private payers shell out top dollar for a drug whose efficacy, in management’s own words, has not yet been established.” The authors also predict that payers will limit access. In fact, Anthem deemed the drug as investigational and not medically necessary, as reported by EndPoints News: “Major Insurer Delivers a Bad Blow to Duchenne Therapies.

While it’s not practical to delve into specific access and reimbursement strategies in this blog post, we do see overall implications for industry:

  • It’s essential to engage stakeholders as early as possible to establish and communicate meaningful measures of efficacy.
  • This sharp criticism comes from pro-industry editors – the debate over value and access is not limited to politicians and payers.
  • Education is needed to ensure a complete understanding of pay-for-performance reimbursement, which may be viable in a single-payer system but may not be practical in the U.S. for some orphan therapies.
  • Moving forward, some orphan drugs may receive the same type of scrutiny as oncology drugs. Namely, does the efficacy demonstrated in clinical trials justify the cost?
Please let us know if you’d like to discuss these implications further with the Rare Collective.
Chris Smith

Chris Smith is the President and CEO of SmithSolve, a communications agency helping clients earn trust, manage risk and break through the noise.

Leave a Reply

close slider

    Connect with the Rare Collective

    Your Email*